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Panobinostat: first global approval
1Springer, Private Bag 65901, Mairangi Bay, 0754, Auckland, New Zealand, dru@adis.com.
Abstract:
Novartis has developed oral and intravenous formulations of panobinostat (Farydak(®)), a histone deacetylase (HDAC) inhibitor, for the treatment of cancer. HDACs have important roles in maintaining chromatin structure and in regulating gene expression, including that of tumour suppressor genes, and thus represent valid targets in the search for cancer therapeutics. Oral panobinostat is approved in the US, as combination therapy with bortezomib and dexamethasone in patients with recurrent multiple myeloma who have received at least two prior treatment regimens, including bortezomib and an immunomodulatory agent. Regulatory submissions have been made for the use of combination therapy with panobinostat in patients with recurrent multiple myeloma in the EU and Japan. Panobinostat is in various stages of clinical development worldwide for a range of haematological and solid tumours. This article summarizes the milestones in the development of panobinostat leading to this first approval for multiple myeloma.
Insights
Panobinostat, a histone deacetylase inhibitor, is approved for treating multiple myeloma. This cancer drug targets gene expression and chromatin structure, offering a new therapeutic option.
Area of Science:
- Oncology
- Pharmacology
- Molecular Biology
Background:
- Histone deacetylases (HDACs) regulate gene expression and chromatin structure, making them targets for cancer therapy.
- Panobinostat is a novel histone deacetylase (HDAC) inhibitor developed by Novartis.
- Dysregulation of HDACs is implicated in the pathogenesis of various cancers, including multiple myeloma.
Purpose of the Study:
- To summarize the key developmental milestones of panobinostat.
- To highlight the regulatory approvals and clinical development of panobinostat for cancer treatment.
- To provide an overview of panobinostat's mechanism of action as an HDAC inhibitor.
Main Methods:
- Review of preclinical and clinical data for panobinostat.
- Analysis of regulatory submission data for panobinostat.
- Summary of ongoing clinical trials investigating panobinostat in various tumor types.
Main Results:
- Oral panobinostat (Farydak®) is approved in the US in combination therapy for recurrent multiple myeloma.
- Regulatory submissions for panobinostat combination therapy in multiple myeloma have been made in the EU and Japan.
- Panobinostat is under investigation in diverse hematological and solid tumors.
Conclusions:
- Panobinostat represents a significant advancement in the treatment of multiple myeloma.
- The development of panobinostat highlights the therapeutic potential of HDAC inhibitors in oncology.
- Panobinostat's broad clinical development indicates its potential utility across a spectrum of cancers.
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